CRISPR inhibition for FSHD will require gene therapy to reach skeletal muscles.

 

Gene therapy can replace a defective gene with a healthy one, introduce a gene to help the body fight disease, or turn off genes that are causing problems. Decades of gene therapy research are paying off.

 

Since August 2017, the U.S. Food and Drug Administration has approved three gene therapy products, the first of their kind.

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Two of these treatments reprogram a patient’s own cells to attack a deadly cancer, and the most recent approved treatment targets a disease caused by mutations in a specific gene.